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Neuroblastoma Treatment (PDQ®)–Patient Version

General Information About Neuroblastoma

Key Points

  • Neuroblastoma is a disease in which malignant (cancer) cells form in neuroblasts (immature nerve tissue) in the adrenal glands, neck, chest, or spinal cord.
  • Neuroblastoma is sometimes caused by a gene mutation (change) passed from the parent to the child.
  • Signs and symptoms of neuroblastoma include a lump in the abdomen, neck, or chest or bone pain.
  • Tests that examine many different body tissues and fluids are used to diagnose neuroblastoma.
  • A biopsy is done to diagnose neuroblastoma.
  • Certain factors affect prognosis (chance of recovery) and treatment options.

Neuroblastoma is a disease in which malignant (cancer) cells form in neuroblasts (immature nerve tissue) in the adrenal glands, neck, chest, or spinal cord.

Neuroblastoma often begins in the nerve tissue of the adrenal glands. There are two adrenal glands, one on top of each kidney in the back of the upper abdomen. The adrenal glands make important hormones that help control heart rate, blood pressure, blood sugar, and the way the body reacts to stress. Neuroblastoma may also begin in nerve tissue in the neck, chest, abdomen, or pelvis.

EnlargeDrawing shows parts of the body where neuroblastoma may be found, including the paraspinal nerve tissue and the adrenal glands. Also shown are the spine and right and left kidney.
Neuroblastoma may be found in the adrenal glands and paraspinal nerve tissue from the neck to the pelvis.

Neuroblastoma most often begins in infancy. It is usually diagnosed between the first month of life and age five years. It is found when the tumor begins to grow and cause signs or symptoms. Sometimes it forms before birth and is found during an ultrasound of the baby.

By the time cancer is diagnosed, it has usually metastasized (spread). Neuroblastoma spreads most often to the lymph nodes, bones, bone marrow, liver, and skin in infants and children. Adolescents may also have metastasis to the lungs and brain.

Neuroblastoma is sometimes caused by a gene mutation (change) passed from the parent to the child.

Gene mutations that increase the risk of neuroblastoma are sometimes inherited (passed from the parent to the child). In children with a gene mutation, neuroblastoma usually occurs at a younger age and more than one tumor may form in the adrenal glands or in the nerve tissue in the neck, chest, abdomen, or pelvis.

Children with certain gene mutations or hereditary (inherited) syndromes should be checked for signs of neuroblastoma until they are 10 years old. The following tests may be used:

  • Abdominal ultrasound: A test in which high-energy sound waves (ultrasound) are bounced off the abdomen and make echoes. The echoes form a picture of the abdomen called a sonogram. The picture can be printed to be looked at later.
  • Urine catecholamine studies: A test in which a urine sample is checked to measure the amount of certain substances, vanillylmandelic acid (VMA) and homovanillic acid (HVA), that are made when catecholamines break down and are released into the urine. A higher than normal amount of VMA or HVA can be a sign of neuroblastoma.
  • Chest x-ray: An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body.

Talk to your child's doctor about how often these tests need to be done.

Signs and symptoms of neuroblastoma include a lump in the abdomen, neck, or chest or bone pain.

The most common signs and symptoms of neuroblastoma are caused by the tumor pressing on nearby tissues as it grows or by cancer spreading to the bone. These and other signs and symptoms may be caused by neuroblastoma or by other conditions.

Check with your child’s doctor if your child has any of the following:

  • Lump in the abdomen, neck, or chest.
  • Bone pain.
  • Swollen stomach and trouble breathing (in infants).
  • Bulging eyes.
  • Dark circles around the eyes ("black eyes").
  • Painless, bluish lumps under the skin (in infants).
  • Weakness or paralysis (loss of ability to move a body part).

Less common signs and symptoms of neuroblastoma include the following:

  • Fever.
  • Shortness of breath.
  • Feeling tired.
  • Easy bruising or bleeding.
  • Petechiae (flat, pinpoint spots under the skin caused by bleeding).
  • High blood pressure.
  • Severe watery diarrhea.
  • Horner syndrome (droopy eyelid, smaller pupil, and less sweating on one side of the face).
  • Jerky muscle movements.
  • Uncontrolled eye movements.

Tests that examine many different body tissues and fluids are used to diagnose neuroblastoma.

The following tests and procedures may be used to diagnose neuroblastoma:

  • Physical exam and health history: An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patient’s health habits and past illnesses and treatments will also be taken.
  • Neurological exam: A series of questions and tests to check the brain, spinal cord, and nerve function. The exam checks a person’s mental status, coordination, ability to walk normally, and how well the muscles, senses, and reflexes work. This may also be called a neuro exam or a neurologic exam.
  • Urine catecholamine studies: A test in which a urine sample is checked to measure the amount of certain substances, vanillylmandelic acid (VMA) and homovanillic acid (HVA), that are made when catecholamines break down and are released into the urine. A higher than normal amount of VMA or HVA can be a sign of neuroblastoma.
  • Blood chemistry studies: A test in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. A higher or lower than normal amount of a substance can be a sign of disease.
  • MIBG scan: A procedure used to find neuroendocrine tumors, such as neuroblastoma. A very small amount of a substance called radioactive MIBG is injected into a vein and travels through the bloodstream. Neuroendocrine tumor cells take up the radioactive MIBG and are detected by a scanner. Scans may be taken over 1-3 days. An iodine solution may be given before or during the test to keep the thyroid gland from absorbing too much of the MIBG. This test is also used to find out how well the tumor is responding to treatment. MIBG is used in high doses to treat neuroblastoma.
  • CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography.
    EnlargeComputed tomography (CT) scan of the abdomen; drawing shows a child lying on a table that slides through the CT scanner, which takes x-ray pictures of the inside of the abdomen.
    Computed tomography (CT) scan of the abdomen. The child lies on a table that slides through the CT scanner, which takes x-ray pictures of the inside of the abdomen.
  • MRI (magnetic resonance imaging) with gadolinium: A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. A substance called gadolinium is injected into a vein. The gadolinium collects around the cancer cells so they show up brighter in the picture. This procedure is also called nuclear magnetic resonance imaging (NMRI).
    EnlargeMagnetic resonance imaging (MRI) of the abdomen; drawing shows a child lying on a table that slides into the MRI scanner, which takes pictures of the inside of the body. The pad on the child’s abdomen helps make the pictures clearer.
    Magnetic resonance imaging (MRI) of the abdomen. The child lies on a table that slides into the MRI scanner, which takes pictures of the inside of the body. The pad on the child’s abdomen helps make the pictures clearer.
  • PET scan (positron emission tomography scan): A procedure to find malignant tumor cells in the body. A small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Malignant tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do.
  • X-ray of the chest or bone: An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body.
  • Ultrasound exam: A procedure in which high-energy sound waves (ultrasound) are bounced off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. The picture can be printed to be looked at later. An ultrasound exam is not done if a CT/MRI has been done.
    EnlargeAbdominal ultrasound; drawing shows a child lying on an exam table during an abdominal ultrasound procedure. A technician is shown pressing a transducer (a device that makes sound waves that bounce off tissues inside the body) against the skin of the abdomen. A computer screen shows a sonogram (picture).
    Abdominal ultrasound. An ultrasound transducer connected to a computer is pressed against the skin of the abdomen. The transducer bounces sound waves off internal organs and tissues to make echoes that form a sonogram (computer picture).

A biopsy is done to diagnose neuroblastoma.

Cells and tissues are removed during a biopsy so they can be viewed under a microscope by a pathologist to check for signs of cancer. The way the biopsy is done depends on where the tumor is in the body. Sometimes the whole tumor is removed at the same time the biopsy is done.

The following tests may be done on the tissue that is removed:

  • Cytogenetic analysis: A laboratory test in which the chromosomes of cells in a sample of tissue are counted and checked for any changes, such as broken, missing, rearranged, or extra chromosomes. Changes in certain chromosomes may be a sign of cancer. Cytogenetic analysis is used to help diagnose cancer, plan treatment, or find out how well treatment is working.
  • Light microscopy: A laboratory test in which cells in a sample of tissue are viewed under regular and high-powered microscopes to look for certain changes in the cells.
  • Immunohistochemistry: A laboratory test that uses antibodies to check for certain antigens (markers) in a sample of a patient’s tissue. The antibodies are usually linked to an enzyme or a fluorescent dye. After the antibodies bind to a specific antigen in the tissue sample, the enzyme or dye is activated, and the antigen can then be seen under a microscope. This type of test is used to help diagnose cancer and to help tell one type of cancer from another type of cancer.
  • MYCN amplification study: A laboratory study in which tumor or bone marrow cells are checked for the level of MYCN. MYCN is important for cell growth. A higher level of MYCN (more than 10 copies of the gene) is called MYCN amplification. Neuroblastoma with MYCN amplification is more likely to spread in the body and less likely to respond to treatment.

Children up to 6 months old may not need a biopsy or surgery to remove the tumor because the tumor may disappear without treatment.

Certain factors affect prognosis (chance of recovery) and treatment options.

The prognosis and treatment options depend on the following:

  • Age at the time of diagnosis.
  • Tumor histology (the shape, function, and structure of the tumor cells).
  • The child's risk group.
  • Whether there are certain changes in the genes.
  • Where in the body the tumor started.
  • The stage of the cancer.
  • How the tumor responds to treatment.
  • How much time passed between diagnosis and when the cancer recurred (for recurrent cancer).

Prognosis and treatment options for neuroblastoma are also affected by tumor biology, which includes the following:

  • The patterns of the tumor cells.
  • How different the tumor cells are from normal cells.
  • How fast the tumor cells are growing.
  • Whether the tumor shows MYCN amplification.
  • Whether the tumor has changes in the ALK gene.

The tumor biology is said to be favorable or unfavorable, depending on these factors. A child with favorable tumor biology has a better chance of recovery.

In some children up to 6 months old, neuroblastoma may disappear without treatment. This is called spontaneous regression. The child is closely watched for signs or symptoms of neuroblastoma. If signs or symptoms occur, treatment may be needed.

Stages of Neuroblastoma

Key Points

  • After neuroblastoma has been diagnosed, tests are done to find out if cancer has spread from where it started to other parts of the body.
  • There are three ways that cancer spreads in the body.
  • Cancer may spread from where it began to other parts of the body.
  • The following stages are used for neuroblastoma:
    • Stage 1
    • Stage 2
    • Stage 3
    • Stage 4
  • Treatment of neuroblastoma is based on risk groups.

After neuroblastoma has been diagnosed, tests are done to find out if cancer has spread from where it started to other parts of the body.

The process used to find out the extent or spread of cancer is called staging. The information gathered from the staging process helps determine the stage of the disease. For neuroblastoma, the stage of disease affects whether the cancer is low risk, intermediate risk, or high risk. It also affects the treatment plan. The results of some tests and procedures used to diagnose neuroblastoma may be used for staging. See the General Information section for a description of these tests and procedures.

The following tests and procedures also may be used to determine the stage:

There are three ways that cancer spreads in the body.

Cancer can spread through tissue, the lymph system, and the blood:

  • Tissue. The cancer spreads from where it began by growing into nearby areas.
  • Lymph system. The cancer spreads from where it began by getting into the lymph system. The cancer travels through the lymph vessels to other parts of the body.
  • Blood. The cancer spreads from where it began by getting into the blood. The cancer travels through the blood vessels to other parts of the body.

Cancer may spread from where it began to other parts of the body.

When cancer spreads to another part of the body, it is called metastasis. Cancer cells break away from where they began (the primary tumor) and travel through the lymph system or blood.

  • Lymph system. The cancer gets into the lymph system, travels through the lymph vessels, and forms a tumor (metastatic tumor) in another part of the body.
  • Blood. The cancer gets into the blood, travels through the blood vessels, and forms a tumor (metastatic tumor) in another part of the body.

The metastatic tumor is the same type of cancer as the primary tumor. For example, if neuroblastoma spreads to the liver, the cancer cells in the liver are actually neuroblastoma cells. The disease is metastatic neuroblastoma, not liver cancer.

Many cancer deaths are caused when cancer moves from the original tumor and spreads to other tissues and organs. This is called metastatic cancer. This animation shows how cancer cells travel from the place in the body where they first formed to other parts of the body.

The following stages are used for neuroblastoma:

Stage 1

In stage 1, the cancer is in only one area and all of the cancer that can be seen is completely removed during surgery.

Stage 2

Stage 2 is divided into stages 2A and 2B.

  • Stage 2A: The cancer is in only one area and all of the cancer that can be seen is not completely removed during surgery.
  • Stage 2B: The cancer is in only one area and all of the cancer that can be seen may or may not be completely removed during surgery. Cancer cells are found in the lymph nodes near the tumor.

Stage 3

In stage 3, one of the following is true:

  • the cancer cannot be completely removed during surgery and has spread from one side of the body to the other side and may also have spread to nearby lymph nodes; or
  • the cancer is on one side of the body and has spread to lymph nodes on the other side of the body; or
  • the cancer is in the middle of the body and has spread to tissues or lymph nodes on both sides of the body, and the cancer cannot be removed by surgery.

Stage 4

Stage 4 is divided into stages 4 and 4S.

  • In stage 4, the cancer has spread to distant lymph nodes or to other parts of the body.
  • In stage 4S, the child is younger than 12 months, and:
    • the cancer has spread to the skin, liver, and/or bone marrow; or
    • the cancer is in only one area and all of the cancer that can be seen may or may not be completely removed during surgery; or
    • cancer cells may be found in the lymph nodes near the tumor.

Treatment of neuroblastoma is based on risk groups.

For many types of cancer, stages are used to plan treatment. For neuroblastoma, treatment depends on the patient's risk group. The stage of neuroblastoma is one factor used to determine risk group. Other factors are age, tumor histology, and tumor biology.

There are three risk groups: low risk, intermediate risk, and high risk.

  • Low-risk and intermediate-risk neuroblastoma have a good chance of being cured.
  • High-risk neuroblastoma may be hard to cure.

Recurrent Neuroblastoma

Recurrent neuroblastoma is cancer that has recurred (come back) after it has been treated. The cancer may come back in the same place or in other parts of the body.

Treatment Option Overview

Key Points

  • There are different types of treatment for patients with neuroblastoma.
  • Children with neuroblastoma should have their treatment planned by a team of doctors who are experts in treating childhood cancer, especially neuroblastoma.
  • Seven types of standard treatment are used:
    • Observation
    • Surgery
    • Radiation therapy
    • Iodine 131-MIBG therapy
    • Chemotherapy
    • High-dose chemotherapy and radiation therapy with stem cell rescue
    • Targeted therapy
  • New types of treatment are being tested in clinical trials.
    • Immunotherapy
  • Treatment for neuroblastoma causes side effects and late effects.
  • Patients may want to think about taking part in a clinical trial.
  • Patients can enter clinical trials before, during, or after starting their cancer treatment.
  • Follow-up tests may be needed.

There are different types of treatment for patients with neuroblastoma.

Different types of treatment are available for patients with neuroblastoma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment.

Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment.

Children with neuroblastoma should have their treatment planned by a team of doctors who are experts in treating childhood cancer, especially neuroblastoma.

Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric health care providers who are experts in treating children with neuroblastoma and who specialize in certain areas of medicine. These may include the following specialists:

Seven types of standard treatment are used:

Observation

Observation is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change.

Surgery

Surgery is used to treat neuroblastoma that has not spread to other parts of the body. As much of the tumor as is safely possible is removed. Lymph nodes are also removed and checked for signs of cancer.

If the tumor cannot be removed, a biopsy may be done instead.

Radiation therapy

Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the cancer.

Iodine 131-MIBG therapy

Iodine 131-MIBG therapy is a treatment with radioactive iodine. The radioactive iodine is given through an intravenous (IV) line and enters the bloodstream which carries radiation directly to tumor cells. Radioactive iodine collects in neuroblastoma cells and kills them with the radiation that is given off. Iodine 131-MIBG therapy is sometimes used to treat high-risk neuroblastoma that comes back after initial treatment.

Chemotherapy

Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy).

The use of two or more anticancer drugs is called combination chemotherapy.

See Drugs Approved for Neuroblastoma for more information.

High-dose chemotherapy and radiation therapy with stem cell rescue

High-dose chemotherapy and radiation therapy are given to kill any cancer cells that may regrow and cause the cancer to come back. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell rescue is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient and are frozen and stored. After the patient completes chemotherapy and radiation therapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells.

Maintenance therapy is given after high-dose chemotherapy and radiation therapy with stem cell rescue for 6 months and includes the following treatments:

See Drugs Approved for Neuroblastoma for more information.

Targeted therapy

Targeted therapy is a type of treatment that uses drugs or other substances to attack cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapies:

  • Monoclonal antibody therapy: This treatment uses antibodies made in the laboratory from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells.

    Pembrolizumab and dinutuximab are monoclonal antibodies being studied to treat neuroblastoma that has come back after treatment or has not responded to treatment.

  • Tyrosine kinase inhibitor therapy: These small-molecule drugs go through the cell membrane and work inside cancer cells to block signals that cancer cells need to grow and divide.

    Crizotinib is a tyrosine kinase inhibitor used to treat neuroblastoma that has come back after treatment. AZD1775 and lorlatinib are tyrosine kinase inhibitors being studied to treat neuroblastoma that has come back after treatment or has not responded to treatment.

  • Histone deacetylase inhibitor therapy: This treatment causes a chemical change that stops cancer cells from growing and dividing.

    Vorinostat is a type of histone deacetylase inhibitor being studied to treat neuroblastoma that has come back after treatment or has not responded to treatment.

New types of treatment are being tested in clinical trials.

This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website.

Immunotherapy

Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. This type of cancer treatment is also called biotherapy or biological therapy.

  • CAR T-cell therapy: The patient's T cells (a type of immune system cell) are changed so that they will attack certain proteins on the surface of cancer cells. T cells are taken from the patient and special receptors are added to their surface in the laboratory. The changed cells are called chimeric antigen receptor (CAR) T cells. The CAR T cells are grown in the laboratory and given to the patient by infusion. The CAR T cells multiply in the patient's blood and attack cancer cells.
    EnlargeCAR T-cell therapy; drawing of blood being removed from a vein in a patient’s arm to get T cells. Also shown is a special receptor called a chimeric antigen receptor (CAR) being made in the laboratory; the gene for CAR is inserted into the T cells and then millions of CAR T cells are grown. Drawing also shows the CAR T cells being given to the patient by infusion and binding to antigens on the cancer cells and killing them.
    CAR T-cell therapy. A type of treatment in which a patient’s T cells (a type of immune cell) are changed in the laboratory so they will bind to cancer cells and kill them. Blood from a vein in the patient’s arm flows through a tube to an apheresis machine (not shown), which removes the white blood cells, including the T cells, and sends the rest of the blood back to the patient. Then, the gene for a special receptor called a chimeric antigen receptor (CAR) is inserted into the T cells in the laboratory. Millions of the CAR T cells are grown in the laboratory and then given to the patient by infusion. The CAR T cells are able to bind to an antigen on the cancer cells and kill them.

    CAR T-cell therapy is being studied to treat neuroblastoma that has come back after treatment or has not responded to treatment.

Treatment for neuroblastoma causes side effects and late effects.

For information about side effects that begin during treatment for cancer, see our Side Effects page.

Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include:

Some late effects may be treated or controlled. It is important to talk with your child's doctors about the effects cancer treatment can have on your child. See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information.

Patients may want to think about taking part in a clinical trial.

For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.

Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.

Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward.

Patients can enter clinical trials before, during, or after starting their cancer treatment.

Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment.

Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.

Follow-up tests may be needed.

Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests.

Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups.

Follow-up tests for patients with neuroblastoma include the following:

Treatment Options for Neuroblastoma

For information about the treatments listed below, see the Treatment Option Overview section.

Low-Risk Neuroblastoma

Treatment of newly diagnosed low-risk neuroblastoma may include the following:

  • Surgery followed by observation.
  • Chemotherapy and surgery, for children with symptoms or children whose tumor has continued to grow and cannot be removed by surgery.
  • Chemotherapy, for certain patients.
  • Observation alone for infants younger than 6 months who have small adrenal tumors or for infants who do not have signs or symptoms of neuroblastoma.
  • Radiation therapy to treat tumors that are causing serious problems and do not respond quickly to chemotherapy or surgery.
  • A clinical trial of treatment based on the tumor's response to treatment and tumor biology.

Intermediate-Risk Neuroblastoma

Treatment of newly diagnosed intermediate-risk neuroblastoma may include the following:

  • Chemotherapy for children with symptoms or to shrink a tumor that cannot be removed by surgery. Surgery may be done after chemotherapy.
  • Surgery alone for infants.
  • Observation alone for infants.
  • Radiation therapy to treat tumors that have continued to grow during treatment with chemotherapy or tumors that cannot be removed by surgery and have continued to grow after treatment with chemotherapy.
  • A clinical trial of treatment based on the tumor's response to treatment and tumor biology.

High-Risk Neuroblastoma

Treatment of newly diagnosed high-risk neuroblastoma may include the following:

Stage 4S Neuroblastoma

There is no standard treatment for stage 4S neuroblastoma but treatment options include the following:

Recurrent Neuroblastoma

Patients First Treated for Low-Risk Neuroblastoma

Treatment for recurrent neuroblastoma that comes back in the area where the cancer first formed may include the following:

Treatment for recurrent neuroblastoma that comes back in other parts of the body or that has not responded to treatment may include the following:

Patients First Treated for Intermediate-Risk Neuroblastoma

Treatment for recurrent neuroblastoma that comes back in the area where the cancer first formed may include the following:

Treatment for recurrent neuroblastoma that comes back in other parts of the body may include the following:

Patients First Treated for High-Risk Neuroblastoma

There is no standard treatment for recurrent neuroblastoma in patients first treated for high-risk neuroblastoma. Treatment may include the following:

Because there is no standard treatment, patients first treated for high-risk neuroblastoma may want to consider a clinical trial. For information about clinical trials, please see the NCI website.

Patients with Recurrent CNS Neuroblastoma

Treatment for neuroblastoma that recurs (comes back) in the central nervous system (CNS; brain and spinal cord) may include the following:

Treatments Being Studied for Progressive/Recurrent Neuroblastoma

Some of the treatments being studied in clinical trials for neuroblastoma that recurs (comes back) or progresses (grows, spreads, or does not respond to treatment) include the following:

Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available.

To Learn More About Neuroblastoma

About This PDQ Summary

About PDQ

Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish.

PDQ is a service of the NCI. The NCI is part of the National Institutes of Health (NIH). NIH is the federal government’s center of biomedical research. The PDQ summaries are based on an independent review of the medical literature. They are not policy statements of the NCI or the NIH.

Purpose of This Summary

This PDQ cancer information summary has current information about the treatment of neuroblastoma. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care.

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Editorial Boards write the PDQ cancer information summaries and keep them up to date. These Boards are made up of experts in cancer treatment and other specialties related to cancer. The summaries are reviewed regularly and changes are made when there is new information. The date on each summary ("Updated") is the date of the most recent change.

The information in this patient summary was taken from the health professional version, which is reviewed regularly and updated as needed, by the PDQ Pediatric Treatment Editorial Board.

Clinical Trial Information

A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.

Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237).

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PDQ® Pediatric Treatment Editorial Board. PDQ Neuroblastoma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/neuroblastoma/patient/neuroblastoma-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389278]

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